Table 1.
Patient and Transplant Characteristics
| Controls (non- cryopreserved graft) |
Cases (cryopreserved graft) |
p-value | |
|---|---|---|---|
| Number | 195 | 52 | |
| Age, median (range) | 22 (4-67) | 21 (5-64) | 0.96 |
| 1 – 17 years | 77 (40%) | 21 (40%) | 0.95 |
| 18 – 39 years | 72 (37%) | 18 (35%) | |
| ≥ 40 years | 46 (24%) | 13 (25%) | |
| Sex, male/female | 115 (59%)/80 (41%) | 22 (42%)/30 (58%) | 0.03 |
| Performance score | 0.72 | ||
| 90 – 100 | 128 (66%) | 31 (60%) | |
| ≤80 | 61 (31%) | 19 (37%) | |
| Not reported | 6 ( 3%) | 2 ( 4%) | |
| Co-morbidity score | 0.55 | ||
| ≤2 | 136 (70%) | 34 (65%) | |
| ≥3 | 59 (30%) | 18 (35%) | |
| Cytomegalovirus serostatus | 0.13 | ||
| Negative | 58 (30%) | 10 (19%) | |
| Positive | 137 (70%) | 42 (81%) | |
| Donor type* | 0.97 | ||
| HLA-matched sibling | 79 (41%) | 21 (41%) | |
| HLA-haploidentical | 25 (13%) | 8 (15%) | |
| HLA-matched unrelated | 64 (33%) | 16 (31%) | |
| HLA-mismatched unrelated | 27 (14%) | 7 (14%) | |
| Donor-recipient ABO match | 0.86 | ||
| Matched | 63 (32%) | 19 (37%) | |
| Minor mismatch | 22 (11%) | 6 (11%) | |
| Major mismatch | 31 (16%) | 6 (11%) | |
| Not reported | 79 (41%) | 21 (41%) | |
| Conditioning regimen** | 0.15 | ||
| Cy + ATG | 59 (30%) | 11 (21%) | |
| Flu + Cy + ATG | 18 (9%) | 6 (12%) | |
| Bu/Cy ± ATG | 4 (2%) | 3 (6%) | |
| Flu + TBI (200 cGy) | 1 (<1%) | 1 (2%) | |
| Cy + ATG + TBI (200 cGy) | 38 (19%) | 8 (15%) | |
| Cy + ATG + TBI (1000 cGy) | 0 | 1 (2%) | |
| Flu + Cy + ATG + TBI (200 cGy) | 55 (28%) | 13 (25%) | |
| Flu + Bu ± ATG | 8 (4%) | 5 (10%) | |
| Flu + melphalan + thiotepa + ATG | 2 (1%) | 2 (4%) | |
| Flu + melphalan | 10 (5%) | 2 (4%) | |
| Graft type | 0.56 | ||
| Bone marrow | 132 (68%) | 33 (64%) | |
| Peripheral blood | 63 (32%) | 19 (36%) | |
| Bone marrow | |||
| Total nucleated cells (x108/kg) Median (IQR) | |||
| Pre-cryopreservation | Not applicable | 3.83 (2.70 – 5.07) N = 19 of 33 |
|
| Infusion | 3.40 (2.45 – 4.57) N = 109 of 132 |
2.63 (1.49 – 3.05) N = 23 of 33 |
0.004 |
| Peripheral blood | |||
| CD34+ (x 106/kg) Median (IQR) |
|||
| Pre-cryopreservation | Not applicable | 7.90 (7.14 – 8.74) N = 15 of 19 |
|
| Infusion | 6.63 (4.78 – 10.97) N = 62 of 63 |
5.38 (3.78 – 10.97) N = 15 of 19 |
0.45 |
| GVHD prophylaxis | 0.07 | ||
| Ex vivo T cell depletion or CD34+ | 18 (10%) | 4 (8%) | |
| Post-transplant Cy + other | 22 (11%) | 6 (12%) | |
| Calcineurin inhibitor + MMF | 21 (11%) | 14 (27%) | |
| Calcineurin inhibitor + MTX | 110 (56%) | 25 (48%) | |
| Calcineurin inhibitor + other | 21 (11%) | 2 (4%) | |
| Other agents | 3 (2%) | 1 (2%) | |
| Interval, diagnosis to transplant | 0.28 | ||
| ≤ 3 months# | 44 (23%) | 8 (15%) | |
| 3 – 6 months╪ | 41 (21%) | 15 (29%) | |
| 7 – 12 months║ | 42 (22%) | 15 (29%) | |
| >12 months# | 68 (35%) | 14 (27%) | |
| Transplant period | 0.16 | ||
| 2013 – 2015 | 103 (53%) | 20 (39%) | |
| 2016 – 2019 | 92 (47%) | 32 (61%) |
Abbreviations:
ABO = blood group A, B, O
Cy = cyclophosphamide; ATG = anti-thymocyte globulin; Flu = fludarabine; TBI = total body irradiation
IQR = inter quartile range
MMF = mycophenolate mofetil; MTX = methotrexate
Donor age, median (range):
Haploidentical: Controls: 32 years (10-65); Cases: 36 years (14-65)
Unrelated: Controls: 27 years (18-59); Cases: 30 years (19-43)
Cyclophosphamide dosing:
Cy + ATG
Cases: Cy dose=200 mg/kg (n=11)
Controls: Cy dose= 200 mg/kg (n=56), Cy dose=120 mg/kg (n=3)
Flu + Cy + ATG
Cases: Cy dose=120 mg/kg (n=5), Cy dose 60 mg/kg (n=1)
Controls Cy dose= 120 mg/kg (n=15), Cy dose 60 mg/kg (n=3)
Bu + Cy
Cases: Cy dose=200 mg/kg (n=1), Cy dose=120 mg/kg (n=2)
Controls Cy dose=200 mg/kg (n=2), Cy dose=120 mg/kg (n=2)
Cy + ATG + TBI (200 cGy)
Cases: Cy dose=200 mg/kg (n=6), Cy dose 29 mg/kg Cases (n=2)
Controls: Cy dose=200 mg/kg (n=22), Cy dose 120 mg/kg (n=1), Cy dose=100 mg/kg (n=4), Cy dose=50 mg/kg (n=2), Cy dose 29 mg/kg (n=8), Cy dose unknown (n=1)
Cy + ATG + TBI (1000 cGy)
Cases: Cy dose=120 mg/kg (n=1)
Flu + Cy + ATG + TBI (200 cGy)
Cases: Cy dose=100 mg/kg (n=4), Cy dose=50 mg/kg (n=4), Cy dose=29 mg/kg (n=5)
Controls: Cy dose=100 mg/kg (n=19), Cy dose=50 mg/kg (n=15), Cy dose=29 mg/kg (n=19)
Interval between diagnosis and transplant
77% were HLA-matched sibling and 23% were HLA-matched or mismatched unrelated donor transplants
55% were HLA-matched sibling, 30% were HLA-matched or mismatched unrelated donor and 14% were HLA-haploidentical transplants
23% were HLA-matched sibling, 59% were HLA-matched or mismatched unrelated donor and 19% were HLA-haploidentical transplants
20% were HLA-matched sibling, 63% were HLA-matched or mismatched unrelated donor and 17% were HLA-haploidentical transplants